KR20180002706A - Smad7 유전자 전달 치료법 - Google Patents

Smad7 유전자 전달 치료법 Download PDF

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Publication number
KR20180002706A
KR20180002706A KR1020177033866A KR20177033866A KR20180002706A KR 20180002706 A KR20180002706 A KR 20180002706A KR 1020177033866 A KR1020177033866 A KR 1020177033866A KR 20177033866 A KR20177033866 A KR 20177033866A KR 20180002706 A KR20180002706 A KR 20180002706A
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South Korea
Prior art keywords
muscle
smad7
raav6
raav
composition
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Ceased
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KR1020177033866A
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English (en)
Korean (ko)
Inventor
부엘 단테스 로져스
폴 그레고리빅
Original Assignee
워싱턴 스테이트 유니버시티
베이커 하트 앤드 다이어비티스 인스티튜트
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Publication of KR20180002706A publication Critical patent/KR20180002706A/ko
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Immunology (AREA)
KR1020177033866A 2015-04-23 2016-04-22 Smad7 유전자 전달 치료법 Ceased KR20180002706A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562151547P 2015-04-23 2015-04-23
US62/151,547 2015-04-23
PCT/US2016/029018 WO2016172608A1 (en) 2015-04-23 2016-04-22 Smad7 gene delivery as a therapeutic

Publications (1)

Publication Number Publication Date
KR20180002706A true KR20180002706A (ko) 2018-01-08

Family

ID=57143535

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020177033866A Ceased KR20180002706A (ko) 2015-04-23 2016-04-22 Smad7 유전자 전달 치료법

Country Status (11)

Country Link
US (1) US11268107B2 (enrdf_load_stackoverflow)
EP (1) EP3285813B1 (enrdf_load_stackoverflow)
JP (1) JP2018519250A (enrdf_load_stackoverflow)
KR (1) KR20180002706A (enrdf_load_stackoverflow)
CN (1) CN107847612A (enrdf_load_stackoverflow)
AU (1) AU2016252887B2 (enrdf_load_stackoverflow)
BR (1) BR112017022621A2 (enrdf_load_stackoverflow)
CA (1) CA2983808C (enrdf_load_stackoverflow)
ES (1) ES2856936T3 (enrdf_load_stackoverflow)
PT (1) PT3285813T (enrdf_load_stackoverflow)
WO (1) WO2016172608A1 (enrdf_load_stackoverflow)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108753943A (zh) * 2018-01-17 2018-11-06 中国医学科学院阜外医院 miR-216a及其靶基因在血管衰老和动脉粥样硬化性心脏病中的应用
CN108498798A (zh) * 2018-04-17 2018-09-07 上海市第六人民医院 一种防治骨骼肌萎缩的治疗药物靶点及其应用
JP2021528639A (ja) * 2018-06-20 2021-10-21 ブリストル−マイヤーズ スクイブ カンパニーBristol−Myers Squibb Company Aav治療の方法
AU2021256895A1 (en) 2020-04-17 2022-11-17 The Trustees Of Columbia University In The City Of New York Methods, compositions and uses thereof for reversing sarcopenia

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS52815B (sr) * 2002-12-20 2013-10-31 Amgen Inc. Vezivna sredstva koja inhibiraju miostatin
WO2004113494A2 (en) 2003-05-21 2004-12-29 Avigen, Inc. Methods for producing preparations of recombinant aav virions substantially free of empty capsids
US7655467B2 (en) * 2003-11-14 2010-02-02 The University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
CN103501803B (zh) * 2010-09-22 2015-12-02 科罗拉多大学董事会 Smad7的治疗应用
US20130136729A1 (en) * 2011-11-11 2013-05-30 University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors
US20140127228A1 (en) * 2012-11-02 2014-05-08 Indiana University School of Medicine Inhibition of tgfbeta signaling to improve muscle function in cancer
FR3004463A1 (fr) * 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective

Also Published As

Publication number Publication date
EP3285813B1 (en) 2020-12-23
PT3285813T (pt) 2021-03-05
EP3285813A1 (en) 2018-02-28
AU2016252887B2 (en) 2021-08-12
ES2856936T3 (es) 2021-09-28
CA2983808C (en) 2022-03-22
CN107847612A (zh) 2018-03-27
CA2983808A1 (en) 2016-10-27
BR112017022621A2 (pt) 2018-07-17
JP2018519250A (ja) 2018-07-19
US11268107B2 (en) 2022-03-08
US20180112232A1 (en) 2018-04-26
AU2016252887A1 (en) 2017-10-26
WO2016172608A1 (en) 2016-10-27
EP3285813A4 (en) 2018-10-10

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